Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseases

Abstract

BackgroundRare diseases are a global public health concern, affecting an estimated 350 million individuals. Only 5% of approximately 7000 known rare diseases have a treatment, and only about half have a patient advocacy organization. Biopharmaceutical companies face complex challenges in developing treatments for rare diseases. Patient advocacy organizations may play a major role by positively influencing research and development, clinical trials, and regulations. Thus, collaboration among patient advocacy organizations and industry is essential to bring new therapeutics to patients.MethodsWe identified an unmet need for guidelines on day-to-day decision-making by rare disease patient advocacy organizations when working with biopharmaceutical partners. We convened an Independent Expert Panel experienced in collaborations between patient advocacy organizations and biopharmaceutical companies (April 2017) to develop consensus guidelines for these relationships. The guidelines were based on an original version by the International Fibrodysplasia Ossificans Progressiva Association (IFOPA). The Expert Panel reviewed and broadened these to be applicable to all patient advocacy organizations. Comments on the draft Guidelines were provided first by Panel participants and subsequently by six independent experts from patient advocacy organizations and industry.ResultsThe Panel comprised four experts from the rare disease community who lead patient advocacy organizations; three leaders who perform advocacy functions within biopharmaceutical companies; and two facilitators, both having leadership experience in rare diseases and industry. The finalized Guidelines consist of four main sections: Identification and Engagement With Companies, Patient Engagement and Patient Privacy, Financial Contributions, and Clinical Trial Communication and Support. The Guidelines address the daily considerations, choices, and consequences of patient advocacy organizations as they engage with biopharmaceutical companies, and offer recommendations for volunteer/paid leaders of the organizations on how to interact in a thoughtful, responsible, ethical way that engenders trust.ConclusionsThese Guidelines recommend best practices and standards for interactions between patient advocacy organizations and industry that will ultimately have a positive effect on the development of novel treatments. Patient advocacy organizations will be provided free access to these Guidelines to help bring clarification to day-to-day decision-making around their interactions, and for use as a living document with the potential for regular revisions and updates.

Financial relationships between patient and consumer representatives and the health industry: A systematic review

“…Stein et al described the development of guidelines on collaboration between rare disease patients’ organizations and the biopharmaceutical industry by an expert panel comprised of leaders from the two groups . Recommendations include seeking funding from multiple companies, a board of directors free from companies’ representatives, and refusing funding for product promotional activities.…”

“…Ideally, funding should be unrestricted or for a specific activity initiated by the organization. Funding sources should be made transparent by the organization and all benefits should be documented . While the Sunshine Act provisions of the Affordable Care Act in the US regarding industry's disclosure of payments to physicians and teaching hospitals do not cover patients or their organizations, there is a case for their expansion to include patients’ organizations, as previously highlighted by McCoy and Karas et al…”

The Horizon of a Therapy for Rare Genetic Diseases: A “Druggable” Future for Fibrodysplasia Ossificans Progressiva

“…In the complex world of research for therapeutic strategies for rare diseases, the support of associations of patients and families is particularly essential, to foster researcher networking and to stimulate the attention of pharmaceutical companies. From this point of view, the efforts made by the FOP patients associations represented by the International FOP Association (IFOPA) in the approaches to pharmaceutical companies is cited as an example and as a basis for guidelines for patient advocacy in the field of rare diseases [ 85 ].…”

Understanding the perceived impact and value of research advocacy initiatives for colorectal cancer

“…Transparency and commitment from both parties should begin on day one." 10 Additionally, it is necessary to have, "reciprocal relationships in which both parties recognize the value of the other." 11 It is also essential to work with advocates from an array of backgrounds and communities to understand the entire patient experience.…”